Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1
Condition(s) studied
Investigational drug(s) / intervention(s)
SRP-1001 for Injection: Single or multiple doses of SRP-1001 by intravenous (IV) infusion
Placebo: Calculated volume to match active treatment by IV infusion
Study summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.
Eligibility
Primary outcome measure(s)
- Number of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study — Part 1: Up to Day 90; Part 2: Up to Day 360
Trial sites (16)
| Facility | City | Region | Status |
|---|---|---|---|
| Liverpool Hospital | Liverpool | New South Wales | |
| Wesley Research Institute | Auchenflower | Queensland | |
| Sunshine Coast University Hospital | Birtinya | Queensland | |
| Alfred Health | Melbourne | Victoria | |
| University of Calgary | Calgary | Alberta | |
| University of Alberta | Edmonton | Alberta | |
| McGill University | Montreal | Quebec | |
| Universitätsklinikum München | München | Germany | |
| Neurologische Universitätsklinik Am Rku | Ulm | Germany | |
| Centro Clinico Nemo Milann | Milan | Italy | |
| Fondazione Policlinico Universitario A. Gemelli | Roma | Italy | |
| Centre For Human Drug Research | Leiden | Netherlands | |
| New Zealand Clinical Research | Auckland | New Zealand | |
| Hospital Universitario Vall d'Hebron | Barcelona | Spain | |
| Hospital Universitario Ramon y Cajal | Madrid | Spain | |
| Hospital Universitari l Politecnic La Fe de Valencia | Valencia | Spain |
More Sarepta Therapeutics, Inc. trials in Spain
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06131983 on ClinicalTrials.gov ↗ ← All trials in Spain